Clinical Trials Directory

Trials / Sponsors / n-Lorem Foundation

n-Lorem Foundation

Academic / Other · 14 registered clinical trials — 4 currently recruiting.

StatusTrialPhaseStarted
Not Yet RecruitingPersonalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuu
Schuurs-Hoeijmakers Syndrome
Phase 1 / Phase 22026-04-01
Not Yet RecruitingPersonalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)
Amyotrophic Lateral Sclerosis
Phase 1 / Phase 22026-03-01
Active Not RecruitingPersonalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation
Dentatorubral-Pallidoluysian Atrophy
Phase 1 / Phase 22025-11-06
Enrolling By InvitationPersonalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charc
Charcot-Marie-Tooth Disease Type 2D
Phase 1 / Phase 22025-10-27
Active Not RecruitingPersonalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Re
Retinal Dystrophy
Phase 1 / Phase 22025-08-28
Active Not RecruitingPersonalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation
Bainbridge-Ropers Syndrome
Phase 1 / Phase 22025-05-19
Active Not RecruitingPersonalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosom
Autosomal Dominant Leukodystrophy
Phase 1 / Phase 22025-03-17
Active Not RecruitingPersonalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With
Neurodevelopmental Disorder With or Without Variable Brain Abnormalities
Phase 1 / Phase 22025-02-24
CompletedPersonalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS
Amyotrophic Lateral Sclerosis
Phase 1 / Phase 22024-11-25
Active Not RecruitingPersonalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation
Dentatorubral-Pallidoluysian Atrophy
Phase 1 / Phase 22024-10-24
Enrolling By InvitationPersonalized Antisense Oligonucleotide for Participants With CHCHD10 ALS
Amyotrophic Lateral Sclerosis
Phase 1 / Phase 22024-10-02
Active Not RecruitingPersonalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS
Amyotrophic Lateral Sclerosis
Phase 1 / Phase 22024-06-24
Active Not RecruitingPersonalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS
Amyotrophic Lateral Sclerosis
Phase 1 / Phase 22024-04-16
Active Not RecruitingPersonalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation
Dentatorubral-Pallidoluysian Atrophy
Phase 1 / Phase 22024-02-21